The conference, set for June 29-July 2, is the largest international conference dedicated entirely to DMD.

Each year, the event attracts at least 500 families from around the world to learn about the latest progress in the fight to end DMD. The workshop follows a January 2017 “Critical Path Innovation Meeting” with the U.S. Food and Drug Administration (FDA).

PPMD organized the 90-minute meeting to explore the role inflammation plays in DMD and how to measure it — including talk about biomarkers across disease stage and different tissue types. Also discussed was the concept that maybe a DMD treatment might not consist of one single dose but a consistent series of treatments over a patient’s lifetime. Officials explored the importance of understanding inflammation pathways at a molecular level so that doctors can treat specific inflammatory processes at the right time.

Informed by this meeting with the FDA, PPMD hopes its pre-conference workshop will cover what’s now known and what’s still to discover about the role of inflammation and immune response in DMD.

“The agenda for this upcoming meeting will include Duchenne experts as well as leaders from other disease areas bringing their expertise and experience to the Duchenne community,” Abby Bronson, PPMD’s senior vice-president of research strategy, said in a press release. “We look forward to better understanding the role that inflammation and immunity play in Duchenne, and we are anxious to continue discussing potential therapeutic options that will help every single person living with this disease.”

The 23rd Annual Connect Conference is partially sponsored by PTC Therapeutics, Santhera Pharmaceuticals, Sarepta Therapeutics, Catabasis Pharmaceuticals and Bamboo Therapeutics (recently acquired by Pfizer). All are all involved in developing treatments for DMD or other muscular dystrophies.

Muscular Dystrophy News

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